A Clinical Study in Male Pediatric Patients With Cerebral X-linked Adrenoleukodystrophy (Cald) to Assess the Effects of MIN-102 Treatment on Disease Progression Prior to Human Stem Cell Transplant (HSCT) (NEXUS)

July 17, 2023 updated by: Minoryx Therapeutics, S.L.

An Open-label, Multicenter Study in Male Pediatric Patients With Cerebral X-linked Adrenoleukodystrophy (Cald) to Assess the Effects of MIN-102 Treatment on Disease Progression Prior to Human Stem Cell Transplant (HSCT)

An Open-Label, multicenter study in male pediatric patients with cerebral x-linked adrenoleukodystrophy (cald) to assess the effects of MIN-102 treatment on disease progression prior to human stem cell transplant (HSCT)

Study Overview

Status

Active, not recruiting

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Estimated)

13

Phase

  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Locations

  • Argentina
    • Buenos Aires
      • Bueno Aires, Buenos Aires, Argentina, B16641NZ
        • Hernan Amartino
  • France
      • Paris, France
        • CHU Kremlin Bicêtre
  • Germany
      • Göttingen, Germany
        • Universitätsmedizin Göttingen Georg-August-Universität
      • Hamburg, Germany
        • Universitätsklinikum Hamburg-Eppendorf Klinik und Poliklinik für Kinder- und Jugendmedizin
  • Spain
      • Barcelona, Spain
        • Hospital Sant Joan de Deu

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

2 years to 12 years (Child)

Accepts Healthy Volunteers

No

Description

Inclusion Criteria:

  • Males aged ≥2 and ≤12 years with a diagnosis of X-linked ALD
  • White matter involvement as determined by cerebral MRI lesions without Gd enhancement at baseline (Population 1), or with Gd enhancement at baseline (Population 2).
  • Major Functional Disabilities (MFD) score of 0.
  • Baseline Loes score >0 and ≤10
  • Gadolinium Intensity Score >2

Exclusion Criteria:

  • Previous HSCT
  • Too far progressed inflammatory brain lesions

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: MIN-102
Drug: MIN-102
Once-daily dosing with a volume specified by the pharmacokineteic specialist to achive the desired plasma exposure. Min-102 Oral suspension, strength 15mg/ml.

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
evaluate whether MIN-102 can halt disease progression of cALD if administered prior to hematopoietic stem-cell transplantation (HSCT), as determined by serial clinical and MRI investigations in pediatric subjects.
Time Frame: 6 months- 2 years
6 months- 2 years
"arrested disease" will be assessed at 24 weeks and at 96 weeks
Time Frame: at 24 weeks and 96 weeks
arrested disease is defined using: Cahnge in NFS from Baseline, Free of MFD and lack of lesion progression on MRI
at 24 weeks and 96 weeks

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Minoryx Therapeutics, S.L.

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

September 13, 2019

Primary Completion (Estimated)

December 31, 2023

Study Completion (Estimated)

December 31, 2023

Study Registration Dates

First Submitted

July 31, 2020

First Submitted That Met QC Criteria

August 24, 2020

First Posted (Actual)

August 27, 2020

Study Record Updates

Last Update Posted (Actual)

July 18, 2023

Last Update Submitted That Met QC Criteria

July 17, 2023

Last Verified

January 1, 2023

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • MT-2-02

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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